Recombinant aav vectors for gene therapy of obesity

C - Chemistry – Metallurgy – 12 – N

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C12N 15/864 (2006.01) A61K 48/00 (2006.01) A61P 3/04 (2006.01) C07K 14/475 (2006.01) C07K 14/52 (2006.01) C07K 14/575 (2006.01) C07K 14/715 (2006.01)

Patent

CA 2410512

Disclosed are methods for the use of cytokine or cytokine receptor-encoding polynucleotides in the creation of transformed host cells and transgenic animals is disclosed. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions comprising polynucleotide sequences that express one or more mammalian cytokines or cytokine receptor polypeptides is described. Also disclosed are methods for the treatment and amelioration of symptoms of a variety of conditions and disorders in an animal, including blindness, retinitis pigmentosa, age-related macular degeneration, obesity, anorexia, and related eating disorders, as well as neurological and musculoskeletal disorders, including for example, amyotrophic lateral sclerosis.

La présente invention concerne des procédés se rapportant à l'utilisation de cytokine ou de polynucléotides codant la cytokine pour la création de cellules hôtes transformées et d'animaux transgéniques. L'invention concerne plus particulièrement l'utilisation de compositions à base de vecteur rAAV (virus adéno-associé recombinant) comprenant des séquences de polynucléotides exprimant au moins une cytokine mammalienne ou des polypeptides récepteurs de cytokine. L'invention concerne enfin des procédures applicables pour le traitement et l'amélioration des symptômes de divers états et troubles affectant l'animal, et notamment la cécité, la rétinite pigmentaire, la dégénérescence maculaire liée à l'âge, l'obésité, l'anorexie, et les troubles liés à l'alimentation, ainsi que des troubles neurologiques et musculo-squelettiques tels que la sclérose latérale amyotrophique.

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